by Madaline Spencer | May 27, 2024
Michael Snape, PhD, Chief Science Officer at AMO Pharma, discusses the efficacy of AMO-02 (tideglusib) for myotonic dystrophy type 1 (DM1). DM1 is a genetic disease affecting the muscles and other body systems. Three forms of DM1 may present: the mild...
by Madaline Spencer | May 23, 2024
Karen Anderson, MD, neuropsychiatrist at Georgetown University, Director of Huntington’s Disease Care, Education, and Research Center, discusses Austedo (deutetrabenazine) to treat patients with Huntington’s disease (HD). HD is a progressive disorder...
by Madaline Spencer | May 21, 2024
Alan Percy, MD, pediatric neurologist at University of Alabama at Birmingham, discusses the DAFFODIL clinical trial, testing the safety and tolerability of trofinetide for the treatment of Rett syndrome. Rett syndrome is a neurodevelopmental condition...
by Peter Ciszewski | May 20, 2024
Anne Pariser, MD, Director of the NCATS’ Office of Rare Diseases Research (ORDR), provides an overview of the ORDR and the research they are involved with. The ORDR is focused on multiple programs to improve the efficacy of rare disease research. Their two...
by Madaline Spencer | May 17, 2024
Doug Treco, PhD, CEO of Inozyme Pharma, discusses ongoing clinical trials for INZ-701, an enzyme replacement therapy for the potential treatment of ENPP1 and ABCC6 Deficiencies. ENPP1 Deficiency and ABCC6 Deficiency are rare metabolic diseases caused by...
